Although the US market is likely more than a year or two away from seeing any commercialized medical products that rely on CRISPR-Cas9 technology, the rapidly developing field has already grabbed the attention of the US Food and Drug Administration (FDA) and other drug regulators.

What is CRISPR?

According to the Broad Institute, CRISPR (pronounced "crisper") stands for Clustered Regularly Interspaced Short Palindromic Repeats, which form the basis for a genome editing technology known as CRISPR-Cas9, which can be programmed to target specific segments of genetic code and edit DNA precisely.

Unlike other genome editing technologies that have entered the clinic, like what Sangamo Therapeutics has been developing, experts say CRISPR-Cas9 is easier.

The potential for such technology to help treat or even cure genetic or other diseases has spurred the creation of a number of different companies, though none of the developing products have begun clinical trials in the US yet.

Regulation and Progress

FDA senior policy adviser Ritu Nalubola explained to attendees at the DIA annual conference in Chicago on Wednesday that the agency is in the early stages of building capacity to regulate treatments that use CRISPR-Cas9 technology, and though they are collaborating with other regulators and working with the National Academies of Science, the cross-border regulation of such treatments seems unlikely.

"Harmonization is not always possible because of the statute," Nalubola said.

And although there is a gene therapy working group within the International Pharmaceutical Regulators Forum that addresses transnational issues, Nalubola said that there's a "lot of research that we can’t oversee," and that there's "a role for further public engagement and addressing best practices."

Kurt von Emster, managing partner of the investment firm Abingworth, which made early investments in the Switzerland-based company CRISPR Therapeutics that he said is now worth about $650 million, noted European regulators have so far been more receptive than FDA.

The European Medicines Agency's Committee for Advanced Therapies has discussed the prospects for CRISPR-Cas9 products. And FDA's Office of Pharmaceutical Quality has an Emerging Technology Team that Nalubola mentioned could help companies with early discussions.

And though the initial hope was that CRISPR-based treatments "would cure one or two diseases, I think that's moved up quite a bit," von Emster said, noting that CRISPR Therapeutics has invested more than $80 million in pre-clinical models and still has its sights on entering clinical trials in the US for the first time before the end of 2017. Massachusetts-based Editas Medicine, which was co-founded by the Broad Institute's Feng Zhang, recently delayed the filing of its IND for its lead CRISPR program.

"This is a technology, not a product," von Emster added, noting that CRISPR Therapeutics decided early to partner with companies like Vertex Pharmaceuticals and Bayer on some projects, though "we decided to keep oncology to ourselves."

He also said the next six months will clarify a lot in the CRISPR space, especially as intellectual property (IP) issues get further ironed out. The US Patent and Trademark Office recently upheld a series of patents granted to the Broad Institute for the CRISPR-Cas9 technology, which will likely be a win for Editas and the Broad Institute.

But Editas, CRISPR Therapeutics and Intellia Therapeutics, among other companies in the CRISPR-Cas9 space, are moving in different directions, von Emster said.

He also told DIA attendees that "no one should think we’re pushing Editas aside," as it's "in our common interests to work together … to overcome the IP situation to get to the task of curing disease."

But in terms of how to approach regulators with what many believe could be breakthrough treatments, companies are just beginning to test the waters.

"We haven't spent a lot of time approaching FDA because we don't know what our product is yet," von Emster said.

Eva Essig, VP of regulatory affairs at Intellia, noted the importance of early technology platform discussions with FDA as such discussions can help with determining which indications are more plausible to bring forward in development.

And both he and Essig said they had their doubts about a small study in mice that made headlines recently on off-target issues with CRISPR, though they think it's important that different approaches are being undertaken.

For now, a lot of the discussion on which treatments will win approval is still hypothetical.

When asked what news headlines will look like a year from now on CRISPR and gene editing, Essig pointed to human trials conducted in China that will probably start seeing early results.